Fortress Biotech announced that the Food and Drug Administration (FDA) has granted Fast Track designation to CUTX-101 (copper histidinate; Cyprium Therapeutics) for the treatment of patients with classic Menkes disease who have not shown significant clinical progression.

Menkes disease is a rare X-linked pediatric disease caused by gene mutations of copper transporter ATP7A; there is currently no FDA-approved treatment. Clinical characteristics of the disease include sparse/depigmented hair, failure to thrive, connective tissue problems, and severe neurological symptoms (i.e., seizures).

CUTX-101, a subcutaneous injectable formulation of copper histidinate, is designed to supplement blood and brain copper levels in patients with Menkes disease. Clinical data from a Phase 1/2 study showed that early initiation with CUTX-101 in patients with Menkes disease showed improvements in neurodevelopmental outcomes and survival. Currently, a Phase 3 study in Menkes disease is underway. 

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This article originally appeared on MPR